从四倍体互补小鼠ıPS细胞到完全多能的患者特异性iPS细胞

Wei Li, Liu Wang, Lei Liu, Xiao-Yang Zhao, F. Zeng, Qi Zhou
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引用次数: 0

摘要

诱导多能干细胞(iPS)在再生医学和药物发现方面具有很大的前景。自从iPS技术发明以来,该领域的中心目标一直是为人类研究和治疗应用获得更安全、更好的iPS细胞。从只有部分多能性的第一代iPS细胞,到能够生殖系传播的iPS细胞,再到目前能够通过四倍体互补产生存活小鼠的iPS细胞,通过小鼠模型积累的知识有望扩展到人类,用于选择和表征完全多能性的人类iPS细胞系。在此,我们回顾了多能诱导多能干细胞的研究进展和策略,讨论了患者特异性诱导多能干细胞在疾病建模和药物发现方面的潜力,并讨论了结合基于诱导多能干细胞的细胞替代疗法的新型基因治疗系统的潜力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
FROM TETRAPLOID-COMPLEMENTING MOUSE ıPS CELLS TO FULLY PLURIPOTENT PATIENT-SPECIFIC iPS CELLS
Induced pluripotent stem (iPS) cells hold great promise for regenerative medicine and drug discovery. Since the invention of iPS technology, a central goal of this field has been to derive safer and better iPS cells for human research and therapeutic applications. From the first generation of iPS cells that were only partially pluripotent, through iPS cells that were capable of germ line transmission, to current iPS cells that can produce viable mice through tetraploid complementation, the accumulating knowledge gained through mouse models is expected to extend to humans for selection and characterization of fully pluripotent human iPS cell lines. Here we review the progress and strategies toward generating fully pluripotent iPS cells, discuss the potential of patient-specific iPS cells in disease modeling and drug discovery, and discuss the potential for novel gene therapy systems combined with iPS cell-based cell replacement therapy.
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